# Proxy Research — Full Description

Source: https://proxyresearch.com/different.html
Last updated: September 2026
Attribution to Proxy Research is requested when this material is quoted.

## What Proxy Research is

Proxy Research Inc. is a rare disease drug repurposing company based in the United States. It uses AI, advanced in vitro models and Real World Data to find new treatments for rare diseases hidden in approved drugs around the world. Proxy evaluates diseases, genetic drivers, therapeutic targets, drugs, modalities and patient populations together, and selects the programs with the highest probability of reaching regulatory approval.

## How Is Proxy Different?

Most companies start with a disease. We start with the entire rare disease landscape.

Traditional rare disease companies often focus on a single disease or technology platform and build everything around that starting point. Proxy systematically evaluates thousands of rare diseases, genetic drivers, therapeutic targets, and approved medicines to identify opportunities where biology, clinical feasibility, and development strategy align. Instead of asking, "How do we develop a treatment for this disease?" we ask, "Which diseases have the highest probability of successful development?"

We optimize for approval, not just discovery.

Finding a promising drug-disease relationship is only the beginning. Many programs fail because of trial design, patient recruitment challenges, safety limitations, regulatory barriers, or commercial realities. Proxy evaluates potential programs through the lens of development success, incorporating clinical, regulatory, and operational factors early in the process to prioritize opportunities that can realistically reach patients.

We use New Approach Methodologies.

Animal studies can't fully anticipate human responses. Differences in target biology, pharmacokinetics, metabolism and off-target effects between species mean many risks only surface in human trials. Proxy uses New Approach Methodologies (NAMs): real tissue, grown from real patients, tested before anyone is exposed to risk. Using patient-derived organoids, we test candidate therapies directly in human tissue, before a compound ever reaches an animal model or a patient.

We focus on mechanisms, not symptoms.

Rare diseases that look similar on the surface may have completely different biological causes. Conversely, diseases that appear unrelated may share common molecular drivers. Proxy integrates genetic, biological, clinical, and pharmacological evidence to identify mechanistic relationships rather than relying on symptom matching alone. The goal is to uncover therapies that address the underlying biology of disease.

Our platform is designed for rare disease reality.

Rare disease development faces unique challenges: small patient populations, fragmented evidence, limited clinical data, and highly heterogeneous disease presentations. Proxy was built specifically to operate in this environment. Our infrastructure, data models, and workflows are designed around rare disease evidence gaps rather than adapted from large-market therapeutic areas.

Every recommendation is traceable.

Scientific and regulatory decisions require transparency. Proxy emphasizes evidence provenance, source tracking, uncertainty identification, and auditability. Rather than treating AI as a black box, we focus on creating evidence-backed outputs that investigators, partners, and regulators can independently review and verify.

We consider the entire development pathway from day one.

Drug repurposing is often approached as a discovery problem. Proxy views it as a development problem. Our process integrates disease biology, target selection, regulatory strategy, natural history evidence, intellectual property considerations, and clinical development planning into a unified framework. This helps ensure that promising candidates are evaluated in the context of the pathway required to bring them to patients.

We are built to enable modern rare disease trial designs.

Conventional randomized controlled trials are often difficult or impossible in very small patient populations. Proxy incorporates natural-history-based approaches, patient-as-own-control strategies, and other rare disease development paradigms to help identify development pathways that can reduce cost, accelerate timelines, and improve feasibility while maintaining scientific rigor.

We search for the right disease, the right target, the right drug, and the right patient.

Many platforms focus on a single dimension of the problem. Proxy evaluates diseases, targets, drugs, modalities, and patient populations together. This multidimensional approach allows us to identify opportunities where multiple factors align, increasing confidence that a therapeutic hypothesis can ultimately translate into clinical success.

We combine AI with regulatory-grade evidence generation.

AI can generate thousands of hypotheses. The challenge is determining which ones matter. Proxy combines computational discovery with structured evidence generation, disease characterization, regulatory planning, and scientific validation to transform hypotheses into development-ready opportunities. Our goal is not simply to discover new ideas, but to create programs capable of advancing toward patients.

## The five stages of Proxy's approach

### 1. Targeting — heterogeneous patient populations lead to heterogeneous results

Proxy starts with the genetics, not the diagnosis. A genetic mutation, not a symptom list, is the starting point. By targeting patients with a single, well-established monogenic cause, Proxy avoids the trap that sinks most rare disease programs: treating biologically distinct conditions as if they were the same disease.

### 2. Selection — most drug candidates fail, and it is bad selection rather than bad luck

Over 300,000 distinct mutations are likely causal for the 6,000+ rare diseases cataloged. Proxy screens approved and investigational medicines to find and formulate the right medicine for the causal mutation, matching the right drug with the right target and the right patients using Real World Data. Candidate sets are narrowed through successive filters — target, protein, potential treatment, safety, pharmacokinetics, pharmacology — each drawing on named public data sources including Open Targets, UniProt, KEGG, the Human Protein Atlas and openFDA.

### 3. Approval — only 5% of repurposed drugs are approved for their new indication

Proxy focuses on drug targets with an Open Targets score above 0.4, which are approved roughly seven times as often. Targets scoring 0.4 and above reach approval in 13.8% of cases against 0.4% below the threshold, with a median 4.7 years to approval against 6.5 years.

### 4. Safety — limited access to biological material creates a bottleneck

Proxy uses New Approach Methodologies (NAMs): real tissue, grown from real patients, tested before anyone is exposed to risk. Using patient-derived organoids, dozens of candidate therapies can be tested directly in relevant tissue before a compound reaches an animal model or a patient. Detecting cardiac liability in a small molecule costs approximately $400,000 over six months in monkeys, against approximately $2,000 over two weeks in microtissues.

### 5. Commercial — a repurposed drug still has to make commercial sense

Repurposing lowers development cost, and regulatory incentives such as Priority Review Vouchers add meaningful value. The core commercial strategy is reformulation: redesigning drugs specifically for children, improving compliance and flattening the pharmacokinetic curve to maximize efficacy while minimizing safety risk. This produces a genuinely better, safer version of the drug that cannot simply be replicated by prescribing the generic off-label. Proxy maintains a 69-drug rare pediatric disease voucher record set covering 11 therapeutic categories, of which 41 programs have disclosed voucher sales.

## Key figures

- 6,000+ rare diseases cataloged, driven by over 300,000 distinct likely-causal mutations.
- 5% of repurposed drugs are approved for their new indication.
- 13.8% approval rate for targets with an Open Targets score of 0.4 and above, against 0.4% below.
- 4.7 median years to approval above the 0.4 threshold, against 6.5 years below.
- $2,000 over two weeks in microtissues to detect cardiac liability in a small molecule, against $400,000 over six months in monkeys.
- 69 programs in the Proxy rare pediatric disease voucher record set, across 11 categories, with 41 disclosed voucher sales.

## Common questions

Q: What does Proxy Research do?
A: Proxy Research is a rare disease drug repurposing company based in the United States. It uses AI, advanced in vitro models and Real World Data to identify new treatments for rare diseases among approved and investigational medicines, then develops those candidates toward regulatory approval.

Q: How is Proxy different from other drug repurposing companies?
A: Most rare disease companies begin with one disease and build everything around it. Proxy begins with the entire rare disease landscape and systematically eliminates opportunities, evaluating diseases, genetic drivers, targets, drugs, modalities and patient populations together, and optimizing for probability of approval rather than discovery alone.

Q: Does Proxy start from a disease or from a portfolio?
A: From a portfolio. Rather than asking how to develop a treatment for a chosen disease, Proxy asks which diseases have the highest probability of successful development.

Q: Is Proxy's AI a black box?
A: No. Proxy emphasizes evidence provenance, source tracking, uncertainty identification and auditability, so that investigators, partners and regulators can independently review and verify the evidence behind each recommendation.

Q: What are New Approach Methodologies, and how does Proxy use them?
A: New Approach Methodologies (NAMs) are non-animal test systems such as patient-derived organoids and human microtissues. Proxy uses them to test candidate therapies in relevant human tissue before a compound reaches an animal model or a patient.

Q: How can I contact Proxy Research?
A: Proxy works with foundations, academic groups and industry partners on rare disease programs. Enquiries can be sent to hello@proxyresearch.com or through the contact form at https://proxyresearch.com/#contact.

## Organization

Proxy Research Inc.
169 Madison Ave STE 38464, New York, NY 10016, United States
Email: hello@proxyresearch.com
LinkedIn: https://www.linkedin.com/company/proxyresearchinc/
Website: https://proxyresearch.com/